Ultrarare inflection point: stakeholders argue for biomarker-based approvals

A meeting convened by the Reagan-Udall Foundation for the FDA on qualifying rare disease biomarkers for accelerated approvals could be an inflection point for patients with ultrarare diseases, their families, biotech companies, and FDA.

The workshop, held on Feb. 21, focused on the use of heparan sulfate measured in cerebrospinal fluid (CSF) to support approval of treatments for neuronal mucopolysaccharidoses (MPS), a set of related ultrarare disorders.  ...